Summary
- Acer received a Complete Response Letter (CRL) from the FDA earlier this week denying approval of EDSIVO and requiring a new clinical trial be conducted.
- Apart from being bad news for the vEDS community in the U.S., the news caused Acer's shares to plummet from the $20 range to the $4 range.
- The company plans to appeal the FDA decision and hopes to work with the vEDS community to try to convince the FDA to reverse, or at least modify, its decision.
- The company will discuss its plans at an investor day next month.
- While the CRL issued on Tuesday was devastating to vEDS patients and Acer shareholders, the Acer story is not over.
Acer Therapeutics Inc. (NASDAQ:ACER) ("Acer" or the "Company") is a pharmaceutical company headquartered in Newton, Massachusetts, focused on the acquisition, development, and commercialization of therapies for serious rare and life-threatening orphan or ultra-orphan diseases with significant unmet medical needs.
Before markets opened on Tuesday, June 25, Acer announced that it had received a Complete Response Letter ("CRL") from the FDA denying approval for its lead drug candidate, EDSIVO, for the treatment of ultra-orphan disease vEDS. Worse still, the FDA is requesting a "new adequate and well-controlled clinical trial" prior to approval. This was seen as a surprise to the Company, the analyst community, the vEDS patient community and most investors. As some have noted it was the FDA itself that had encouraged Acer to get EDSIVO approved in the U.S. in the first place (approximately 4 years ago), as there was no approved or effective treatment available to treat patients suffering from this ultra-orphan disease.
The type of trial being requested by the FDA in its CRL would take many years (if not a decade or more) to complete and would be a very difficult type of trial for which to recruit patients. Not only is the population of vEDS patients very small, but what vEDS patient would want to volunteer to participate in a clinical trial that could go on for years knowing that he or she had a 50% chance of being given a placebo!
If the FDA is not persuaded to change its mind and Acer is required to conduct such a trial before approval of EDSIVO, I doubt that it will do so.
Quite frankly, the CRL issued by the FDA and its trial requirement is a disaster for the U.S. vEDS community, Acer Therapeutics and its shareholders. Given how well-known the safety and long-term data from celiprolol (EDSIVO) is relating to the treatment of vEDS, and given the fact that there is no approved or effective treatment option for vEDS patients in the U.S, the FDA decision appears to be short-sighted.
I read a post this morning on StockTwits (a site which is generally focused only on investing and trading of stocks) which was very moving and sums up the human cost to the FDA's decision.
